Sangamo Fabry disease gene therapy study (ST-920-201)

Brief description of study

Fabry disease is caused by mutations in the GLA gene. The GLA gene encodes the lysosomal enzyme alpha-galactosidase A, or a-Gal A for short. When a-Gal A is missing or deficient, a substance called globotriaoslyceramide, or Gb3 for short, and related materials build up in the vessels, nerves, tissues, and organs throughout the body. This buildup may cause progressive kidney failure, heart disease, cerebrovascular disease, skin lesions, and other abnormalities. The current standard of care for treating Fabry disease is enzyme replacement therapy (ERT) or pharmacological chaperone therapy. ERT uses manufactured versions of a-Gal A, including Fabrazyme®. Treatment with ERT has been shown to reduce the deposits of Gb3. However, limitations to ERT include the need for life-long treatment, development of neutralizing antibodies that impact how effective the treatment is for the person with Fabry disease, and the inconvenience of frequent infusions. This study will test to see if doctors can use a process called “gene therapy” to treat Fabry disease. Gene therapy is a technique that aims to replace the broken gene with a working copy. The gene will be delivered into your cells using a vector. A vector is a virus that has been modified to include a gene. It has also been changed so it cannot reproduce itself; therefore, the vector can deliver the gene into cells, but it can no longer cause a disease or infection. The virus used in this study is called an adeno-associated virus (AAV). This type of virus is very good at entering cells. The study drug is an AAV vector carrying the GLA gene. Think of the vector as a car and the gene as the passenger. The car (the vector) will drive the passenger (the GLA gene) where it needs to go (the liver cells) and drop it off there. Although the virus has already been administered to humans, this is the first study in which ST-920 (the virus plus the GLA gene) will be given to humans, but it has been studied in animals. The main purpose of this research study is to see if receiving ST-920 is safe and well tolerated. The potential effect of ST- 920 for improving your Fabry disease will also be evaluated. ST- 920 is considered a new investigational medicine which is not approved by the FDA, however the FDA has approved this study to take place.

Clinical Study Identifier: s18-02063 Identifier: NCT04046224

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